Our Studies Underway
Research Collaboration Towards New Treatments
With world-class expertise, a culture of cross-disciplinary collaboration, and a proven track record of successfully bringing therapies from the lab to patients, our physicians and researchers are leading the field in developing treatments and cures for childhood cancers and rare diseases.
We continue to push the boundaries of science to increase the understanding of pediatric cancers and other diseases that can be treated with blood and marrow transplantation (BMT). Together, we challenge the status quo and help to change the practice of medicine.
Our research is focused in three areas; finding treatments and cures for the incurable; how to make transplant more successful and improving the long-term health and outcomes for our patients.
Our Research and Education Efforts
Our faculty and staff are committed to research and education. Physicians and scientists of the Pediatric BMT Program actively engage in research to increase the understanding of pediatric cancers and other diseases that can be treated with blood and marrow transplantation. Combining our renowned classroom teaching with our pioneering research creates benefits for the next generation of health care providers, our patients and their families, and the general public. Please contact us if you have questions or want more information. Patients may become involved in our research and education efforts by participating in a clinical trial.
BMT Survivor Study
One of our largest projects is the Bone Marrow Transplant Survivor Study with more than 3,500 transplant survivors. It involves a questionnaire that covers late medical effects and quality of life. It will provide a tremendous amount of information regarding the medical conditions BMT survivors face. The study will be one of the largest and most comprehensive ever performed. It is being conducted in partnership with investigators from the City of Hope National Medical Center in California.
Cord Blood Research
We are one of six BMT centers approved for the National Cord Blood Transplant Study, funded by the National Heart, Lung and Blood Institute of the National Institute of Health. The purpose of this four-year study is to critically evaluate techniques of collecting, storing and transplanting umbilical cord blood, and evaluate long-term survival rates of cord blood transplant patients. University of Minnesota scientists are also investigating the transplantation of two partially matched cord blood collections from different donors to increase the number of stem cells transplanted to adult patients. Another approach for adult patients involves using a lower-dose chemotherapy and radiation treatment prior to cord blood transplant for patients who cannot tolerate the higher doses used in conventional transplants. Both studies show promising results with high rates of engraftment and a low incidence of graft-versus-host disease.
Childhood Cancer Survivorship
Identifies and characterizes factors associated with adverse long-term health and psychosocial outcomes among survivors of childhood and adolescent cancer in order to develop and test intervention strategies to improve the overall long-term survival and quality of life of successfully treated patients.
Epidemiologic Research Program
Studies the potential genetic and/or environmental influences that determine a child's risk of developing cancer. The program director is currently leading the effort to establish a Childhood Cancer Research Network, a North American pediatric cancer registry that will facilitate nontherapeutic research.
Fanconi Anemia Treatment Options
University of Minnesota scientists continue to lead the nation in several areas of research to improve Fanconi Anemia (FA) patient outcomes, including: use of preimplantation genetic diagnosis to create a healthy HLA-matched sibling donor; multipotent adult stem cell therapy to treat other tissues of the body rather than just the marrow; hematopoietic stem cell gene correction in FA; and immune reconstitution to reduce the risk of infection after BMT.
Hematology/Genetic Disease Program
Investigates new strategies using new cellular therapies generated in the Stem Cell Biology and Immune-Based Therapy Programs to treat patients with genetic or metabolic diseases (e.g., Fanconi Anemia, Krabbe Disease, Adrenoleukodystrophy). Researchers are working to develop a new transplantation protocol for individuals with high-risk, inherited genetic or metabolic disorders.
Immunotherapy to Prevent Relapse
University of Minnesota researchers are incorporating immunotherapy into the transplant process. The goal is to use these therapies pre- or post-transplant to enhance the effectiveness of the transplant and improve patient outcomes. Examples of immunotherapies include the use of Natural Killer (NK) cells, Interleukin-2 (IL-2) and anti-tumor vaccines. Another approach studied by Paul Orchard, M.D., uses gene therapy to target the destruction of immune cells by "suicide genes" to better control graft-versus-host disease.
Michael Verneris, M.D. and Jeffrey Miller, M.D. are evaluating NK cell infusions to fight cancer for patients with advanced acute myelogenous leukemia. A patient's own NK cells recognize a cancer tumor as the body's own and do not destroy it. We have found, however, that donor NK cells see the tumor cells as foreign, offering a potentially effective therapy. Future plans call for combining NK cell infusions with the best available stem cell transplant strategy to induce the most optimum clinical response. Ultimately, better control of the immune system holds great promise for improving patient outcomes.
University of Minnesota Masonic Children's Hospital Blood and Marrow Transplant Services is a world leader in the field of hematopoietic stem cell transplantation to treat metabolic disorders. The International Storage Disease Collaborative Study Group, an international group of researchers and clinicians dedicated to advancing treatment for inherited metabolic storage diseases through collaborative studies and data sharing, is based at the University of Minnesota. Scientists from the University of Minnesota's Stem Cell Institute, Comprehensive Cancer Center and Medical School provide additional expertise in the latest technology in treating these diseases.
Molecular Cancer Biology Program
Investigates molecular mechanisms of pathogenesis of childhood cancers that will increase understanding of the underlying biology of the disease and help in developing better treatments.
Phase I Experimental Therapeutics Program
Develops and tests new treatment strategies for children with cancer resistant to conventional therapy, or who are at high risk for treatment failure.
Quality of Life
Researchers are working to improve the quality of life after treatment. Of interest is the late effects patients are experiencing related to fertility, growth and development, potential chronic organ damage and risk of second cancers. Analyzing this information will help to guide treatment improvements and identify ways to help survivors cope with long-term complications.
Stem Cell Biology Program
Conducts research into the use of stem cells obtained from umbilical cord blood and adult tissues and develops novel approaches for treating children with tissue injury resulting from cancer or cancer treatment. The program recently discovered new ways to use umbilical cord blood that lowered relapse rates and nearly tripled survival rates. In addition, the program has been working with new stem cell transplant options designed to make transplants readily available for nearly all patients.
Unrelated Donor Transplantation
Researchers continue to make progress and are currently working to reduce side effects when using marrow from unrelated donors. They are also investigating new drugs that may reduce graft-vs-host disease (GVHD), a potentially major complication of BMT in which the donor cells recognize the body's own cells as foreign and trigger the immune system to attack those cells.
"They are so excited because not only are they learning how to treat and hopefully cure EB, but they are learning other things. Dr. Wagner mentioned that they may be able to help regenerate new skin for burn victims in the future. There is so much more potential here."